**: Pharmaceutical Intelligence Headlines***
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In 2023, the healthcare sector has experienced many compelling innovations that have not only improved the way disease** is done, but also offer new hope and possibilities for global health.
In addition to the milestone of the first CRISPR being approved, Cell has made significant progress in a wide range of diseases. By manipulating the patient's own cells, scientists are able to develop personalized protocols for diseases such as cancer, immune system disorders, and more. This customized approach makes ** more effective and reduces the likelihood of adverse effects.
The success of antibody-drug conjugates (ADCs) has also brought a new dawn to oncology**. ADCs combine antibodies and drug molecules to precisely target cancer cells and reduce damage to healthy cells. This revolutionary** approach improves the survival rate of cancer patients and significantly improves their quality of life.
The success of large amounts of RNA has also opened up new avenues. By intervening in gene expression, RNA can lead to a range of diseases, including hereditary diseases, metabolic diseases, and more. This precise method provides a new solution to some diseases that have plagued human beings for a long time.
These innovative breakthroughs across the healthcare industry are providing more choice for patients, while also driving medical professionals around the world to be more actively engaged in scientific research and clinical practice. With the continuous advancement of technology and the accumulation of medical knowledge, human efforts in defeating disease will continue to achieve remarkable results, bringing more hope for the future of healthcare.
Will the pharmaceutical field continue to ride the momentum of innovation in 2023 in 2024 and achieve more ambitious breakthroughs in new fields? Executives from several biopharma and biotech companies have their own views on the outlook for 2024. How do they see the development of new technologies translating into industry change in 2024?
Drug Innovation Trends
Bloom at multiple points
More antibody drugs are on the market
Tahi Ahmadi, Executive Vice President, Genmab: The development of new models such as antibody-drug conjugates, bispecific antibodies, and cells and genes** has taken a larger share of the research pipeline. In 2023, these modal drugs have received a large number of FDA approvals. The focus on these new diseases is indicative of efforts to address previously difficult-to-treat diseases, including advanced cancers and rare diseases. With hundreds of antibodies in preclinical and clinical development, the number of antibody drugs is expected to continue to increase, and the importance of antibody drugs will become more apparent, both in pipeline and approved products.
The development of cells and genes** will become more streamlined
Michelle Fraser, Head of Cells and Genes** at Revvity: More than 500 new cells and genes** enter clinical trials each year, and the time to market is significantly accelerated. At the same time, the FDA's regulatory initiatives to promote these initiatives are expected to usher in a new era of standardization. This includes streamlining manufacturing processes, integrating Critical Quanlity Attribute (CQA) testing protocols, improving patient identification and enrollment methods, and the development of related diagnostics.
Interpretation: The tide of advanced ** approval is coming. The FDA plays an important and active role in driving these developments, especially in the area of standardization. Expectations for standardization include streamlining manufacturing processes, consolidating testing protocols, refining methods for patient identification and recruitment, and the development of companion diagnostics technologies. These measures herald a more efficient future of cells and genes**, providing patients with faster, safer** options.
Delivery technology takes "delivery" genes to the next level
lexeo therapeutics ceo r.Nolan Townsend: Genes are growing rapidly and are expanding into new areas, such as cardiovascular disease, including cardiomyopathy with genetic markers for disease. Innovations in delivery technologies will continue to support this shift, especially a**adeno-associated virus) technology. At the same time, there has been a significant shift in the regulatory landscape for genetics** and cardiovascular disease, with the FDA increasingly accepting biomarkers as a key endpoint of study, not just survival.
Interpretation: Innovative delivery technology will promote the broader indication field of gene ** grace. In addition to this, the strategies of regulatory agencies have also contributed to the adoption of genes**, especially clinical endpoints, which are no longer paranoid about survival and are now allowing for greater adherence to biomarkers. This illustrates the change in the criteria for evaluating the effect, with a greater focus on biological characteristics, which improves the accurate assessment of the effect. This trend brings new challenges and opportunities for the future of genetics** and cardiovascular diseases**.
The research and development of anti-aging drugs will be more precise
Carrie Strom, Senior Vice President and President, Allergan Aesthetics, ABBVIE: Aging is an "indication" with a 100% prevalence that everyone is exempt from. As a result, there will be more focus on how to address the root causes of aging to maintain energy, mobility, and youthful appearance. This will give rise to the next generation of research and ** that incorporates the root causes of aging, rather than just addressing the signs and symptoms of the aging process to support the goals of health and longevity.
Interpretation: The medical field will see more research on anti-aging drugs, and it will directly point to the root cause of aging, rather than making superficial articles. Rather than just coping with the superficial symptoms of the aging process, there will be a greater focus on research and ** to address the root causes of aging. There will be a next generation of research and research, which may include understanding the aging process at the molecular and cellular levels, and developing more precise, individualized approaches.
The next generation of mRNA is on the horizon
Stefan Merlo, Vice President of Commercial Development, CSL Seqirus: Vaccine innovation will be one of the key concerns in the pharmaceutical field in 2024, and the emergence and application of emerging technologies will play a key role in the growth of innovative vaccines, such as self-amplifying mRNA (SA-mRNA). There is always a need for flexible and effective vaccine technology, and the COVID-19 pandemic has given us enough warning and inspiration in this regard. mRNA technology can facilitate rapid and scalable vaccine development and manufacturing. SA-mRNA technology offers the potential to provide a stronger immune response and a longer duration of protection while using much lower doses than traditional mRNA vaccines.
Interpretation: SA-mRNA (self-amplifying mRNA) is a variant of mRNA technology with self-replicating properties. Unlike traditional mRNA vaccines, SA-mRNA contains not only information encoding vaccine proteins, but also genetic elements capable of replicating themselves. This allows more vaccine proteins to be produced within the cell, which increases the strength and persistence of the immune response. There are several reasons why sa-mRNA technology is becoming increasingly important:
Self-replicating feature:SA-mRNA has the ability to replicate itself, producing more vaccine proteins within the cell. This self-increasing property allows for a strong immune response to be triggered even at lower doses, increasing the efficacy of the vaccine.
Quick response and flexibility:SA-mRNA technology is responsive and flexible in vaccine development. In the face of novel pathogens, scientists are able to design, synthesize, and produce SA-mRNA vaccines more quickly to respond to new health threats in a short period of time.
Lower doses:Due to the self-replicating nature of SA-mRNA technology, similar or better results can be achieved at lower doses than traditional mRNA vaccines. This is of great significance for the large-scale production and distribution of vaccines.
Long-term immune protection:The self-increasing properties also help provide longer immune protection. The immune system is activated over a long period of time due to the continuous production of vaccine proteins in cells, allowing the immune response to persist for a period of time, thus increasing the durability of the vaccine.
In summary, SA-mRNA technology is becoming increasingly important in the field of vaccines, and its self-replicating properties give vaccines stronger and longer-lasting immune effects, providing a more flexible and efficient tool for dealing with new pathogens.
The field of obesity will evolve rapidly
Jayson Dallas, CEO of Rivus Pharmaceuticals: Obesity is a major driver of metabolic disease, and in 2024, the morbidity and mortality caused by obesity will remain higher than that of all cancers combined. In 2024, one will continue to see the development and growth of GLP-1 and other incretins** as the broader obesity market continues to grow. When people consider the next generation of metabolic disease methods, there is a need to focus on molecular targets with novel and alternative mechanisms, including those that safely increase metabolic rate. Machine learning and artificial intelligence are expected to play a more critical role in this area to identify new small molecule drug candidates in a faster and more efficient way.
Interpretation: Although Eli Lilly's Zepbound and Novo Nordisk's Wegovy have become the standard in the field of medicine, there is still a huge share of this market for latecomers to continue to explore. Many pharmaceutical giants, including Roche, are also actively expanding their arms war, hoping to make huge profits in the obesity market. However, whether the new generation of first-class drugs can break through several traditional targets of GLP-1, GIP and Glucagon, and achieve a breakthrough in pharmacology will become the key to who can emerge in the future. Especially in the competition for the development of small molecule oral drugs, who can take the lead in breaking the monopoly of peptide drugs is also the focus of the industry. Artificial intelligence and machine learning will play an important role in the development of small molecule drugs.
Investment in antimicrobial resistance (AMR) will increase
Margaret Borys, Senior Vice President and Chief Commercial Officer, Shionogi: Investments in antivirals and antibiotics will pay off. COVID-19, influenza and respiratory syncytial virus are not going away and could still wreak havoc across the globe. People need to actively respond to the huge threat of antimicrobial resistance (AMR). According to the United Nations, AMR could cause up to 10 million deaths by 2050. And new viral threats can emerge at any time, leading to future epidemics. This highlights the importance of AMR research.
Interpretation: The increased resistance of microorganisms (bacteria, fungi, viruses and parasites) to drugs will lead to the ineffectiveness of antibiotics, antiviral drugs, etc. Antimicrobial resistance is an important issue in global public health, which can lead to difficult infectious diseases, increased healthcare costs, and a serious health threat to society. Therefore, the research, prevention and management of antimicrobial resistance are considered to be urgent and important.
R&D projects will pay more attention to the voices of patients
Roger Palframan, U.S. Head of Research, UCB: Patient Centricity means that it's critical for drug developers to listen to and interact with patients who are seriously ill and to use relevant disease models to delve deeper into the pathways that lead to disease. Patient life experiences must be carefully considered in the earliest stages of development, ensuring that the drugs being developed positively improve their lives, and focusing on addressing the underlying biological causes of these diseases. This means bringing the research team closer together with patients and caregivers.
Interpretation: In the early stage of research, the concept of paying more attention to patients is gradually formed. This patient-centered approach means that researchers recognize the importance of listening and interacting with patients and their caregivers. The patient's life experience must be fully taken into account, which facilitates more targeted research and development.
Antineoplastic drugs**
Breakthroughs will be made
The importance of early diagnosis of cancer
Mohit Manrao, Senior Vice President and Head of Oncology USA, AstraZeneca: The earlier cancer is detected and addressed, the closer it is to a better outcome. In 2024, it is expected that people will continue to turn to early detection and intervention to gain proactive action on the road to cancer by improving access to cancer screening, and starting earlier**.
Progression will be made in refractory cancers
Guoliang Yu, CEO and Chairman of Apollomics: The pharmaceutical field has long been plagued by the continuous challenges of refractory and refractory cancers. For example, although PD(L)-1 inhibitors have been shown to be effective in many cases of NSCLC, they do not respond to some forms of NSCLC. In addition, resistance develops in patients who initially respond to some**.
To address these issues, efforts are being made to deal with these refractory cancers. With a strong focus on cutting-edge technology platforms and a strong drug development portfolio, these R&D developments are expected to make substantial progress in 2024. Integrating sound mechanisms and scientific principles in clinical trials, exploring single and combined, and making a major leap forward towards overcoming difficult cancer types.
Interpretation: In 2024, the research and development prospects for refractory cancers show a positive trend. Research is more focused on gaining a deeper understanding of the biology of these cancers, especially their complex driving mechanisms. It is expected to see the emergence of more innovative drugs and methods, especially in the field of immunology. With the progress of science and technology, the research and development of new drugs and the development of more accurate methods, it is expected to create a new situation for the development of refractory cancers. At the same time, strategies for combining ** may be explored to enhance the ** effect.
Regulatory reform is more flexible
Emil Kakkis, CEO of Ultragenyx Pharmaceutical: The entire pharmaceutical industry is pushing the FDA to provide more guidance and support to achieve genetics that promote rare and ultra-rare diseases**, which will remain a theme in 2024.
FDA leadership is interested in making this happen, but the pharmaceutical industry needs to see aspirations translated into practice. Bipartisan support in Congress will allow the FDA to adjust its policies to keep pace with innovation. But to make the change, the FDA needs to shift to a more flexible approach, such as putting patients with degenerative rare diseases at the center of decision-making.
Interpretation: In the eyes of pharmaceutical companies, the pace of FDA reform should be greater to keep up with the rapid advancement**. Of course, this is just a family statement, not a full consideration from the perspective of the FDA. For example, the emphasis on the need to put those with degenerative rare diseases at the heart of decision-making, while this is in line with the patient-centric philosophy, there is also some "coercion of patients to order the FDA".
ref.parrish, m. pharm**oice’s crystal ball: what’s next in drug innovation and clinical trials. pharm**oice. 11. 01. 2024.
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